AB1717-PARE DERMATOMYOSITIS BURDEN OF DISEASE MANIFESTATIONS AND QOL IMPACT: PATIENT-REPORTED SURVEY RESULTS
Authors: L. Christopher Stine, J. Feldman, T. Smith, P. Mudd, J. Paik
Journal: Annals of the Rheumatic Diseases (2023)

<h3>Background</h3> Dermatomyositis (DM) is a rare, multi-system idiopathic inflammatory myopathy characterized by skin rash and muscle weakness that is often accompanied by pain and fatigue. Other manifestations include interstitial lung disease and calcinosis. Malignancy is also highly associated with DM. The profound negative clinical impacts of DM on patients' quality of life (QoL) are not yet well-characterized in the literature.<sup>[1]</sup> <h3>Objectives</h3> To capture patient perspectives on the impact of DM on QoL (physical function, mental health, personal relationships, and work productivity), most bothersome symptoms (MBS), and severity of symptom burden in adults. <h3>Methods</h3> A 60-question survey was developed via sponsor-conducted focus groups and adaptions from existing patient-reported outcome (PRO) tools (e.g., WPAI-GH, SLE-FAMILY, and PROMIS). After IRB approval, members of The Myositis Association (~4000 individuals with DM) were invited to complete the online survey (eligibility criteria: 18 – 75 years of age, self-reported DM diagnosis, and current DM symptoms persistent for ≥ 1 year). Responses were collected per the following Likert scales: DM severity rated from "mild" to "very severe" (4-point scale), overall health rated from "poor" to "excellent" (5-point scale), life impact questions rated from "never" to "always" or "not at all" to "a great deal" (5-point scales), and severity of symptoms rated from "never experienced" to "very severe" (6-point scale). <h3>Results</h3> Of 195 respondents (97% from US), the median age was 57 years, 88% female, and 82% White. 53% and 35% had experienced DM symptoms for 3 to 10 years or &gt; 10 years, respectively. 92% were currently receiving medication(s) to manage DM symptoms. The 5 MBS reported were muscle weakness (44%, n = 86), fatigue (43%, n = 84), muscle pain (30%, n = 59), itchy skin (24%, n = 46), and skin rash (19%, n = 37). A majority of respondents indicated DM had at least a "moderate" negative impact on the following domains: ability "to do the things they enjoy" (83%, n = 162), ability to engage in social activities (75%, n = 146), sexual desire and ability to engage in physically intimate relationships (64%, n = 125), relationships outside of family (57%, n = 112), and relationships with family (51%, n = 100). A majority reported negative impacts on mental health (83%, n = 162), defined as having felt "anxious or nervous" or "down, depressed or hopeless" at least "sometimes" due to DM. Almost half reported being "usually" or "always" worried about symptom flares (49%, n = 96) and about their ability to carry out daily activities (42%, n = 82). DM symptom severity was associated with negative impact on overall health. "Fair" or "poor" health was reported by 50% (n = 98) of those experiencing "moderate" DM symptoms, by 85% (n = 27) of those with "severe" symptoms, and by 100% (n = 7) of those with "very severe" symptoms. All with "very severe" DM responded "usually" or "always" to questions about DM limiting their ability to "do the things they enjoy", concern about symptoms "getting worse", and ability to "carry out daily activities." DM symptoms hindered physical functioning, with 63% and 65% of respondents, overall, reporting that DM limited their ability to climb stairs and perform usual daily activities. DM also affected respondents' ability to work; nearly half were unemployed (47%, n = 92) and had to change their employment status due to DM (49%, n = 95), while 24% (n = 47) were receiving disability assistance. Of respondents who were working, 69% (n = 77) reported that DM negatively impacted their career/career choices. <h3>Conclusion</h3> The survey results highlight the marked physical and psychosocial burden of DM in adults and the impact of disease severity on the ability to perform daily activities, mental health, interpersonal relationships, and work productivity. These results underscore the unmet need for new treatment options to ameliorate the progressive and debilitating consequences of DM and further inform PRO-focused research in DM. <h3>Reference</h3> [1]Alexanderson et al. J Rheumatol. 2014;41(3):581-92. <h3>Acknowledgements:</h3> NIL. <h3>Disclosure of Interests</h3> Lisa Christopher Stine Consultant of: Janssen, Boehringer-Ingelheim, Mallinckrodt, EMD-Serono, Allogene, Argenx, Jolie Feldman Shareholder of: Roivant Sciences, Employee of: Roivant Sciences, Priovant Therapeutics, Taryn Smith Employee of: Priovant Therapeutics, Paul Mudd Shareholder of: Roivant Sciences, Priovant Therapeutics, Employee of: Priovant Therapeutics, Julie Paik Speakers bureau: Argenx, Consultant of: Argenx, Pfizer, Kezar Life Sciences, Roivant Sciences, EMD-Serono, Alexion, Guidepoint, Grant/research support from: Pfizer, EMD-Serono, Kezar Life Sciences, Alexion.